The growing role of PROs in clinical research
In the pursuit of optimal patient-centred care, patient-reported outcomes (PROs) are gaining prominence in the regulatory landscape. PROs are data collected directly from patients regarding their symptoms, functional status and overall quality of life. They provide valuable insights into patients’ perspectives, preferences and treatment experiences that may not be fully captured by traditional clinical endpoints alone.
In recent years, regulatory agencies have shown an increasing interest in utilising PRO data in clinical trials to inform decision-making. Including PRO endpoints in clinical trial designs allows researchers to measure patients’ experiences and tailor treatments to better meet their needs.
PROs can make a difference when unmet needs are high: the case of ovarian cancer
When effective and tolerable management options are scarce, PROs can help identify treatment benefits beyond traditional clinical endpoints, such as improved symptom management and enhanced patient satisfaction. They may help gain access to therapeutic approaches that, whilst not superior to the current standard of care in terms of efficacy, may provide similar benefits with a lesser toll on quality of life.
We can look at ovarian cancer as a key example of the crucial relevance of harnessing the power of PROs. It affects hundreds of thousands of women worldwide, with incidence and mortality rates predicted to rise significantly in the next few decades. Despite important progress in diagnosis and management, unmet needs remain, urging the search for new and innovative treatment options. Promising therapies are emerging, and the importance of PROs is gaining recognition in the regulatory environment, paving the way for more patient-centred care.
Current investigations in ovarian cancer include PROs among key study outcomes
Despite the challenges, the landscape of ovarian cancer treatment is evolving. Researchers and pharmaceutical companies are investigating novel approaches to pharmacological management, including targeted therapies, immunotherapies, combination treatments and cutting-edge technologies such as CAR-T cells and antibody-drug conjugates.
In recent years, both the EMA and the FDA have officially encouraged and recommended the inclusion of PROs in clinical trial designs, particularly in the oncology space. This acknowledgement by two key regulatory bodies has also meant a wider recognition in the scientific/medical community and among pharmaceutical companies, encouraging them to work more closely with the patients they aim to treat and to listen more attentively to their needs, expectations and hopes. Seminal studies in ovarian cancer have included PROs among their assessments, and a set of ovarian cancer-specific PROs called MOST (Measure of Ovarian Symptoms and Treatment concerns) has been developed to assess tolerability in women with ovarian cancer, either receiving palliative care or following first-line treatment (MOST-S26).
Cognite recently spoke with healthcare providers specialised in gynaecological malignancies across several European Countries. The topic of PROs in ovarian cancer was spontaneously raised during our conversations: one oncologist mentioned that ‘PROs could support clinical decisions when efficacy and safety of a treatment are comparable with standard of care’, whilst another physician, also a researcher and principal investigator in several studies, recognised that ‘PROs are acquiring an increasing importance in clinical trials’.
Can Medical Affairs mediate the dialogue between industry and regulatory bodies?
The Medical Affairs function has the opportunity to play a vital role in fostering a transparent, fruitful collaboration between their companies and regulatory bodies at global and local levels, with the ultimate goal of granting access to novel, cutting-edge treatments with established benefit/risk ratios to as many eligible patients as possible. As the nature of PROs is deeply connected with subjectivity and emotion, as well as with strict clinical evaluations, a communication strategy that finds the perfect balance between these facets becomes paramount and may concretely change the future of entire patient populations.
By spearheading collaborative efforts among researchers, healthcare professionals, regulatory agencies and patients, Medical Affairs function can help establish their company as leaders worth following in key therapy areas and ultimately overcome the challenges posed by ovarian cancer and other diseases that have so far proven difficult to manage satisfactorily.
If you have any questions or would like to hear more from our Medical team, reach out to Joanne Pearson via email at joanne.pearson@cognite.co.